Chugai's Bispecific Antibody "ACE910" for the Treatment of Hemophilia A Designated as a Breakthrough Therapy by the US FDA

4.9.2015 05:00 | Business Wire

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Chugai Pharmaceutical Co., Ltd. [Head Office: Chuo-ku, Tokyo; Chairman &
CEO: Osamu Nagayama] (hereafter, “Chugai”) announced today that the US
Food and Drug Administration (FDA) has granted the Breakthrough Therapy
Designation to “ACE910,” for the prophylactic treatment of people who
are 12 years or older with hemophilia A with factor VIII inhibitors.

“We are very pleased that the FDA has granted Breakthrough Therapy
Designation to ACE910,” said Chugai’s Director and Executive Vice
President, Dr. Yutaka Tanaka. “We believe with its new mode of action,
ACE910 will contribute to the patients by offering new choice of
treatment options.”

Hemophilia A is a disease presenting repeated severe bleeding symptoms.
In this disease, the blood coagulation reaction does not proceed
normally due to the deficiency or functional disorder of coagulation
factor VIII. Although the regular factor VIII replacement therapy has
been widely used to prevent bleeding, this intravenous treatment may
develop anti-factor VIII neutralizing antibodies (inhibitors) which
result in reduced effectiveness. Under these circumstances, hemophilia A
is a disease with high unmet medical needs.

ACE910 is a bispecific antibody created using Chugai’s proprietary
antibody engineering technologies for the purpose of mimicking the
function of blood coagulation factor VIII, in order to provide a novel
concept for the treatment of hemophilia A. The Japanese phase I / II
clinical study is currently being conducted to investigate the safety
and exploratory prophylactic efficacy of ACE910 for preventing bleeding
with the once-weekly subcutaneous injection of ACE910 in patients both
with and without inhibitors.
(Chugai’s press release dated June 23,
2015 on the interim results of this study:http://www.chugai-pharm.co.jp/english/news/detail/20150623083000.html)

The breakthrough therapy designation was adopted as part of the FDA
Safety and Innovation Act (FDASIA) enacted in July 2012 aiming at
expediting the development and review of drugs for the treatment of
severe or life-threatening diseases or symptoms. The designation for
ACE910 was based on the domestic phase I / II clinical study conducted
with Japanese and Caucasian healthy volunteers as well as Japanese
hemophilia A patients. It is the third such designation for a drug
candidate developed by Chugai after alectinib (for patients with ALK-
positive non-small cell lung cancer whose disease progressed on
crizotinib therapy) and tocilizumab (systemic sclerosis).

Development rights for regions other than Japan, Taiwan, and Korea were
licensed out to F. Hoffman-La Roche, Ltd. [Headquarters: Basel,
Switzerland; CEO: Severin Schwan] (hereafter, “Roche”) (Roche
development code: RG6013). Chugai and Roche plan to initiate a phase III
global study in patients with inhibitors by the end of 2015 and a phase
III global study in patients without inhibitors in 2016. Additionally, a
trial in pediatric patients with hemophilia A is planned to commence in
2016.

Based on its business philosophy of “innovation all for the patients,”
Chugai will work with Roche towards the marketing applications of ACE910
in countries around the world in order to provide the new treatment
option to patients and healthcare professionals as soon as possible.

[Reference]About ACE910
ACE910 is a bispecific
antibody created by Chugai, and substitutes for the function of blood
coagulation factor VIII, which is deficient or lowered in hemophilia A
patients, by promoting factor IXa-catalyzed factor X activation as
factor VIII does1) 2). ACE910 is expected to prevent bleeding
irrespective of the presence of inhibitors because ACE910 has a
different molecular structure from FVIII, and the interim analysis of
the domestic clinical studies showed the favorable results by
once-a-week subcutaneous administration of ACE910.

About Breakthrough Therapy
The breakthrough therapy
designation was adopted as part of the FDA Safety and Innovation Act
(FDASIA) enacted in July 2012 aiming at expediting the development and
review of drugs for the treatment of severe or life-threatening diseases
or symptoms. In order to grant breakthrough therapy designation,
preliminary clinical evidence is required demonstrating that the drug
may have substantial improvement on at least one clinically significant
endpoint over existing therapies. Although breakthrough therapy
designation differs from other FDA systems that expedite the development
and review of pharmaceutical products, it is a system that conveys all
of the benefits of the fast track designation.

About Chugai
Chugai Pharmaceutical is one of Japan’s leading
research-based pharmaceutical companies with strengths in biotechnology
products. Chugai, based in Tokyo, specializes in prescription
pharmaceuticals and is listed on the 1st section of the Tokyo Stock
Exchange. As an important member of the Roche Group, Chugai is actively
involved in R&D activities in Japan and abroad. Specifically, Chugai is
working to develop innovative products which may satisfy the unmet
medical needs, mainly focusing on the oncology area.
In Japan,
Chugai’s research facilities in Gotemba and Kamakura are collaborating
to develop new pharmaceuticals and laboratories in Ukima are conducting
research for technology development for industrial production. Overseas,
Chugai Pharmabody Research based in Singapore is engaged in research
focusing on the generation of novel antibody drugs by utilizing Chugai’s
proprietary innovative antibody engineering technologies. Chugai Pharma
USA and Chugai Pharma Marketing are engaged in clinical development
activities in the United States and Europe.
The consolidated
revenue in 2014 of Chugai totaled 461.1 billion yen and the operating
income was 77.3 billion yen (IFRS Core basis).
Additional
information is available on the internet at http://www.chugai-pharm.co.jp/english.